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AUCD’s Maternal and Child Health Engagement supports the Autism CARES Programs, including LEND and DBP Programs.

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AUCD’s Public Health Team works to create health promotion and public health efforts that include people with disabilities.

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Project

Study to Investigate Long-Term Safety and Efficacy of GS-100

Center:

TX-LoneStar, LEND

Fiscal Year:

2024

Contact Information:

Holly Kathryn Harris
[email protected]
832-824-8369

Project Description:

A Phase 1/2/3 Open-Label, Single Arm, Dose-Finding Study to Investigate Long-Term Safety and Efficacy of GS-100, an Adeno-Associated Virus Serotype 9(AA V9) Vector-Mediated Gene Transfer of Human NGLY, in Patients with NGLY1 Deficiency Grace Science Foundation Industry Funded Co-Investigator, Suter (PI) 01/2024 – present

Keyword(s):

Core Function(s):

Performing Direct and/or Demonstration Services, Performing Research or Evaluation, Continuing Education/Community Training

Area of Emphasis

Other

Target Audience:

Adults with Disabilities, Children/Adolescents with Disabilities/SHCN

Unserved or Under-served Populations:

Other

Primary Target Audience Geographic Descriptor:

National

Funding Source: